Clinical Trials 101

Clinical trials are research studies conducted in people to assess whether treatments are effective and safe. Clinical research is the only way to turn promising science into treatments for people. Without clinical research and human volunteers, we cannot create better treatment and prevention strategies for the disease.
Clinical trials on ALD/AMN are designed to test new ways to:

Phases of drug clinical trials

Laboratory-based research, or pre-clinical research, takes place before any testing in humans is performed. These studies provide a scientific basis for a potential drug or treatment that may be safe and effective on people. Once a drug has been developed using preclinical research, the drug must complete a three-phase clinical trial process (Phase I, II, and III) before the FDA can approve the drug for public’s use. The drugs must perform well in each phase to progress to the next one. It often takes 10 to 15 years or more to complete all 3 phases of clinical trials.

Placebo-Controlled Trials

Most ALD/AMN patients and caregivers want to do everything they can to help find new treatments for the disease. The idea of trying an experimental therapy is often regarded positively, despite knowing that risks may be present. However, some people have misgivings when they learn that some participants in a trial may receive a placebo rather than the drug being tested.

Why is clinical research so important to finding a cure?

People with ALD who enroll in a clinical trial are contributing to improved health care for everyone with the disease. Even when the results of a trial are negative, we learn more about the disease as well as how to look for more promising new treatments. The more researchers and the public know about ALD, the greater the chance of finding a treatment that prevent or reverses the symptoms of ALD.

Because ALD is a rare hereditary disease, it is very important to enroll people who have the disease as well as family members who may be carriers for the disease. Recruiting and retaining trial participants is crucial to better understand the long-term effects of a treatment on disease symptoms and outcome. When you take part in a clinical trial, you add to the knowledge about ALD and help to accelerate the process to find potential intervention and preventative strategies.

What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

We recommend visiting the Center for Information and Study on Clinical Research Participation (CISCRP) webpage for additional educational material on clinical trials. CISCRP is an independent non-profit organization dedicated to educating and informing the public and patients about clinical research. CISCRP also provides information and resources to help research and health professionals better serve their patients and study volunteers. CISCRP has excellent educational videos on their website which illustrate some best practices for clinical trial participants. Click here to see the videos.